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Parkinson's disease stopped in aimal model... /
Parkinson's disease stopped in animal model Molecular 'tweezers' break up toxic aggregations of proteins
"We call this unique mechanism
'process-specific,' rather than the common
protein-specific inhibition," Bitan added, meaning
the compound only attacked the targeted aggregates
and nothing else.
The researchers next tried their tweezers in a
living animal, the zebrafish, a tropical freshwater
fish commonly found in aquariums. The zebrafish is a
popular animal for research because it is easily
manipulated genetically, develops rapidly and is
transparent, making the measurement of biological
Using a transgenic zebrafish model for Parkinson's
disease, the researchers added CLR01 and used
fluorescent proteins to track the tweezer's effect
on the aggregations. They found that, just as in
cell cultures, CLR01 prevented α-synuclein
aggregation and neuronal death, thus stopping the
progression of the disorder in the living animal
Being able to prevent α-synuclein from aggregating,
prevent toxicity and break up existing aggregates is
a very encouraging result, but still, at the end of
the day, "we've only stopped Parkinson's in
zebrafish," Bronstein said.
"Nonetheless," he said, "all of these benefits of
CLR01 were found without any evidence of toxicity.
And taken together, CLR01 holds great promise as a
new drug that can slow or stop the progression of
Parkinson's and related disorders. This takes us one
step closer to a cure."
The researchers are already studying CLR01 in a
mouse model of Parkinson's and say they hope this
will lead to human clinical trials.
Other authors of the study included Shubhangi
Prabhudesai, Sharmistha Sinha, Aida Attar, Aswani
Kotagiri, Arthur G. Fitzmaurice, Ravi Lakshmanan,
Magdalena I. Ivanova, Joseph A. Loo and Mark Stahl,
all of UCLA, and Frank-Gerrit Klärner and Thomas
Schrader of the University of Duisburg–Essen in